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Aurora magazine

Can Parkinson's be prevented with gene therapy?

In Parkinson's and in some forms of dementia, toxic aggregates that destroy neural circuits are created in the brain. Osaka University researchers are testing preventive gene therapy. The first studies concern only the guinea pigs; if all goes well, we will also move on to human experimentation. Although Parkinson's disease is widespread, the causes are still unclear.

Existing therapies are only symptomatic in nature and there is no real cure. Also for this reason, researchers are looking for preventive therapies that reduce the onset of the disease in people at risk. Japanese gene therapy affects a protein called alpha-synuclein, which aggregates into so-called Lewy bodies. According to the researchers, avoiding the formation of bodies could prevent or even reverse the disease. To test this theory, they tested it in vitro and on animal models.

The researchers created a section of genetic material that corresponds to the alpha-synuclein. At this point they stabilized the genetic fragments, modifying them so that they prevented the formation of the protein itself and therefore of the aggregates. To obtain the best possible result, they tested different variants of modified genetic fragments. In this way they have come to reduce protein levels by about 80% in vitro.

Later, scientists tested genetic fragments on animal models with Parkinson's. They came to the brain without major problems, without the need for chemicals to carry them. The treated mice showed the first improvements within 27 days of therapy.